This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853), will fund a $705,402 research project to develop a gene therapy treatment for Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD), a rare genetic metabolic disorder. The project aims to use patient-derived induced pluripotent stem cell (iPSC) neurons and a mouse model to study the efficacy and safety...
This federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports the development and testing of adeno-associated virus (AAV) gene therapy constructs for the treatment of sialidosis, a rare and fatal lysosomal storage disorder. The $679,207 award to the University of Massachusetts Medical School will fund preclinical studies in mouse and sheep...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $226,500 Project Grant (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to the University of Georgia Research Foundation, Inc. to characterize and treat peripheral nervous system dysfunction in Sanfilippo syndrome, a rare genetic disorder also known as mucopolysaccharidosis type IIIA.
The key objectives of this 2-year project include: 1) Developing novel cellular models...
This $124,246 federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) supports the development of advanced CRISPR-based gene editing strategies to correct common ALS-causing mutations in the SOD1 gene. The key objectives are to:
Develop AAV-mediated base editing approaches to correct the SOD1 A5V mutation in vitro.
Develop AAV-mediated...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $374,349 Project Grant (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to the University of North Carolina at Chapel Hill (UNC-CH) to develop and optimize a new animal model for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The project will leverage CRISPR technology to generate transgenic mice exhibiting key hallmarks of ALS/FTD, such as TDP-43...
This $1,359,618 federal Project Grant award was provided by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. The grant aims to comprehensively characterize the protein poly-ADP-ribosylation (PARylation) landscape in the context of the C9ORF72 genetic mutation, which is the most common cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)....
This $564,186 federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) seeks to investigate how a specific genetic mutation (PACS2E209K) associated with a neurodevelopmental disorder known as PACS2 syndrome impacts neuronal metabolism and neurotransmission. The research, to be conducted at the University of Pittsburgh, will utilize advanced live-cell...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $151,600 project grant under CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders to the Philadelphia College of Osteopathic Medicine (PCOM) to generate and characterize a Cre-Lox regulated transgenic zebrafish model of spinal and bulbar muscular atrophy (SBMA). This rare neuromuscular disorder, also known as Kennedy's Disease, affects men and currently has no cure or therapy....
The National Institute on Aging (NIA), under the Aging Research Federal Grant Program (CFDA 93.866), awarded a $1,698,323 Project Grant to the University of California, San Diego (UCSD) to develop and validate a mouse model for Alzheimer's disease in Down syndrome (DS-AD). The key objectives are to:
Demonstrate feasibility and scale of use, and internally validate the mouse model by documenting molecular, cellular, and synaptic age-related phenotypes characteristic of DS-AD and dependent on...
This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) aims to develop a new therapy for neurodevelopmental mitochondrial disorders. The $105,625 award, with a period of performance from August 1, 2024 to July 31, 2026, will support research to: 1) analyze how a specific succinyl-CoA precursor is metabolized in a mouse model of pyruvate dehydrogenase...